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PDUFA VI goals letter released

Aug 22, 2016

On Friday, July 15, the Food and Drug Administration published the performance goals and procedures letter for the Prescription Drug User Fee Act (PDUFA) reauthorization for fiscal years 2018–2022, known as PDUFA VI. As it currently stands, PDUFA’s legislative authority is set to expire in September 2017.

PDUFA, which requires reauthorization every five years, was established in 1992 to supplement the human and financial resources available to the FDA to review and approve new drug and biologic applications. As such, the program is a key priority for Astellas and the broader biopharmaceutical industry.

Over the past year, members of the Astellas team have worked closely with counterparts at the Pharmaceutical Research and Manufacturers of America (PhRMA) and Biotechnology Innovation Organization (BIO) to help inform the scope of the industry’s positions.

Under the PDUFA process, PhRMA and BIO represent the industry in negotiations with FDA to help shape a set of policy priorities for the user fee program. The results are reflected in the performance goals letter. Specifically, the agreement:

Facilitates science-based integration of the patient perspective into the development and regulatory review of innovative medicines.

Enhances the FDA’s access to the tools, processes and expertise necessary to keep pace with the latest scientific advances in drug development and regulation.

Helps accelerate the development and availability of new medicines to patients while providing scientific and regulatory predictability that will foster continued biopharmaceutical innovation.

Ensures that FDA can hire and retain a strong scientific and medical workforce to advance its public health mission.

Of the many priorities in PDUFA VI, a few stand out. Building on efforts in PDUFA V, the performance goals would help further integrate the patient perspective into drug development and regulatory decision-making. For example, FDA will publish guidance on patient data collection and impact – allowing for the first time the possibility of patient-reported information to be included in a drug’s label.

As part of efforts to enhance the scientific tools that FDA uses to make regulatory decisions, the agreement would advance the use of real-world evidence in decisions regarding both safety and effectiveness through workshops, new guidance and pilot studies.

In addition, the agreement would establish for the first time hiring goals for the program to ensure that FDA review divisions are fully staffed and the necessary expertise is available to the agency.

Following the publication of the goals letter, PhRMA President and CEO Stephen Ubl released a statement, saying, “With the successful conclusion of the PDUFA VI technical negotiations between the FDA and the biopharmaceutical industry, PhRMA looks forward to working with Congress, the administration, patient and medical provider groups, the FDA and all stakeholders to ensure timely reauthorization of this important program.”

Next Steps

On August 15, 2016, the FDA will hold a public hearing on PDUFA VI to discuss the performance goals letter. The agency will accept written comments for 30 days. FDA will consider such public views and comments and revise the recommendations as necessary.

The PDUFA reauthorization process will then shift to Capitol Hill for final authorization. Congress will create legislation that likely will include additional policy initiatives that were not part of the final negotiated package.

There are many consistencies between the PDUFA VI performance goals letter and the Senate’s consideration of the “21st Century Cures Act” which was passed by the House of Representatives last year.

Stay tuned for a deeper dive into the performance goals letter in an upcoming article on ChangeTogether.com.

Astellas Patient Advocacy is a function within Corporate Affairs at Astellas that focuses on creating, building and maintaining third-party relationships. We serve as a conduit between Astellas and external stakeholders to help improve patient outcomes, improve access issues and address patients’ unmet needs head on.